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Drugs. 2016 Sep;76(13):1281-1291. doi: 10.1007/s40265-016-0622-z.
Efmoroctocog alfa (Elocta(®), Eloctate(®), Eloctate™), a first-in-class recombinant factor VIII-Fc fusion protein (rFVIIIFc), has an extended half-life compared with conventional factor VIII (FVIII) preparations, including recombinant FVIII (rFVIII) products. It is approved for the treatment and prophylaxis of bleeding in patients with haemophilia A in multiple countries worldwide. Data accumulated from pivotal phase III studies (A-LONG in adults and adolescents aged ≥12 years; Kids A-LONG in children aged <12 years) and their ongoing extension study (ASPIRE) have demonstrated the long-term effectiveness of efmoroctocog alfa for the treatment of acute bleeding episodes, perioperative management and routine prophylaxis in previously treated males with severe haemophilia A. Among patients on individualized efmoroctocog alfa prophylaxis who had previously received FVIII prophylaxis, all but one of those aged ≥12 years and three-quarters of those aged <12 years reduced their injection frequency compared with their pre-study regimen. FVIII replacement therapy with efmoroctocog alfa was generally well tolerated in previously treated patients, with no evidence of increased immunogenicity. The safety and efficacy of FVIII replacement therapy with efmoroctocog alfa in previously untreated males aged <6 years with severe haemophilia A are currently being evaluated. Although there are no direct, head-to-head studies, the available clinical trial evidence indicates that efmoroctocog alfa provides an effective alternative to conventional FVIII preparations (including rFVIIIs) for the management of haemophilia A. Moreover, by reducing the frequency of injections required, it has the potential to reduce treatment burden, and hence improve adherence to prophylaxis.
依非洛科奥塔（Elocta(®)、Eloctate(®)、Eloctate™），一种新型的重组因子 VIII-Fc 融合蛋白（rFVIIIFc），与传统因子 VIII（FVIII）制剂相比，半衰期更长，包括重组 FVIII（rFVIII）产品。它在全球多个国家被批准用于治疗和预防血友病 A 患者的出血。来自关键的 III 期研究（A-LONG 研究用于年龄≥12 岁的成人和青少年；Kids A-LONG 研究用于年龄<12 岁的儿童）及其正在进行的扩展研究（ASPIRE）的数据表明，依非洛科奥塔长期用于治疗先前治疗的重度血友病 A 男性的急性出血发作、围手术期管理和常规预防，具有疗效。在接受个体化依非洛科奥塔预防治疗的患者中，所有年龄≥12 岁的患者中有除一人外，其余患者的注射频率均低于研究前的方案，而所有年龄<12 岁的患者中有四分之三的患者注射频率降低。依非洛科奥塔替代治疗在先前接受过治疗的患者中通常耐受性良好，无免疫原性增加的证据。目前正在评估依非洛科奥塔替代治疗在未曾接受过治疗的年龄<6 岁的重度血友病 A 男性中的安全性和疗效。尽管没有直接的头对头研究，但现有临床试验证据表明，依非洛科奥塔为血友病 A 的管理提供了一种有效替代传统 FVIII 制剂（包括 rFVIII）的选择。此外，通过减少所需的注射频率，它有可能减轻治疗负担，从而提高预防治疗的依从性。
